Safety and Efficacy of Immunomodulator Drug Thalidomide in Multi-transfused Thalassemia Patients

March 15, 2025 updated by: Muhammad Aamir Latif

Safety and Efficacy of Immunomodulator Drug Thalidomide in Multi-transfused Thalassemia Patients: A Single Center Experience From South Punjab, Pakistan

This study aimed to determine the safety and efficacy of thalidomide in multi-transfused thalassemic children.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

Given the significant burden of transfusion dependency, an effective and safe inducer is needed that could improve quality of life and reduce the healthcare costs associated with frequent transfusions and iron chelation therapy. If this study could establish the safety of thalidomide in terms of teratogenicity along with its efficacy, it would be a great support to multi-transfused thalassemic children.

Study Type

Interventional

Enrollment (Actual)

121

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Pakistan
    • Punjab
      • Multan, Punjab, Pakistan, 66000
        • The Children's Hospital & Institute of Child Health

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Children of both genders
  • Aged 2-5 years
  • With transfusion-dependent thalassemia (TDT)
  • Whose parents/guardians showed willingness to adhere to follow-up visits

Exclusion Criteria:

  • Children with uncontrolled infections
  • Significant hepatic or renal dysfunction
  • Malignancy
  • Known contraindications to thalidomide

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Thalidomide group
Thalidomide was administered at a dose of 1.5 mg/kg/day, and all patients continued standard iron chelation therapy and other supportive treatments as per institutional protocols.
Drug: Thalidomide
Thalidomide was administered at a dose of 1.5 mg/kg/day, and all patients continued standard iron chelation therapy and other supportive treatments as per institutional protocols.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Efficacy of treatment
Time Frame: 6 months
Efficacy was categorized as excellent response showing an increase in hemoglobin by 1-2 g/dL or freedom from blood transfusions, partial response as an increase in hemoglobin <1 g/dL or a decrease in transfusion frequency, or no response if there was no increase in hemoglobin or an increase in transfusion requirement within three months.
6 months
Safety of treatment
Time Frame: 6 months
Treatment was considered safe if no adverse events were documented based on clinical assessment and laboratory findings.
6 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Muhammad Aamir Latif

Investigators

  • Principal Investigator: Muhammad Aslam, FCPS, Children's Hospital and institute of Child Health Multan, Punjab, Pakistan
  • Principal Investigator: Sara Rubab, FCPS, Children's Hospital and institute of Child Health Multan, Punjab, Pakistan

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 1, 2023

Primary Completion (Actual)

December 31, 2024

Study Completion (Actual)

December 31, 2024

Study Registration Dates

First Submitted

March 15, 2025

First Submitted That Met QC Criteria

March 15, 2025

First Posted (Actual)

March 25, 2025

Study Record Updates

Last Update Posted (Actual)

March 25, 2025

Last Update Submitted That Met QC Criteria

March 15, 2025

Last Verified

March 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • DRASLAMCHMULTAN

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

Data can be shared on a reasonable request.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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